A Phase 1, Dose Escalation Trial of RP-A601 in Subjects With PKP2 Variant-Mediated Arrhythmogenic Cardiomyopathy (PKP2-ACM)
A Phase 1 Dose Escalation Trial Evaluating an Intravenously Administered Recombinant Adeno-Associated Virus Serotype rh.74 (AAVrh.74) Vector Containing the Human Plakophilin-2a (PKP2a) Coding Sequence (RP-A601; AAVrh.74-PKP2a) in Subjects With Arrhythmogenic Cardiomyopathy Arising From Pathogenic PKP2 Variants (PKP2-ACM)
Rocket Pharmaceuticals Inc.
9 participants
Aug 29, 2023
INTERVENTIONAL
Conditions
Summary
This Phase 1 dose escalation trial will assess the safety and preliminary efficacy of a single dose intravenous infusion of RP-A601 in high-risk adult patients with PKP2-ACM.
Eligibility
Inclusion Criteria7
- Male or female ≥18 years at the time of signing the informed consent
- Capable and willing to provide signed informed consent
- Clinical diagnosis of ACM as defined by the 2010 revised Task Force Criteria (TFC)
- Documentation of a pathogenic or likely pathogenic truncating variant in PKP2
- History of Implantable Cardioverter-Defibrillator (ICD) implantation ≥6 months prior to enrollment
- PVC frequency ≥500 per 24 hours by ambulatory rhythm monitoring
- Left ventricular ejection fraction by echocardiogram or CMR ≥50%
Exclusion Criteria5
- Anti-AAVrh.74 capsid neutralizing antibody titer of \>1:40
- Cardiomyopathy related to a genetic etiology other than PKP2 truncating variant
- Previous participation in a study of gene transfer or gene editing
- Severe Right ventricular (RV) dysfunction
- New York Heart Association (NYHA) Class IV heart failure.
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Interventions
RP-A601 is a recombinant viral vector composed of an AAV serotype rh.74 (AAVrh.74) capsid encapsulating the transgene, human plakophilin 2 (PKP2), transcript variant 2a (PKP2a)
Locations(3)
View Full Details on ClinicalTrials.gov
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NCT05885412